Browsing by Author "Santos, Tiago Miguel Dourado"
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- MicroRNAs in pathology and as therapeutic target in gene therapy?Publication . Santos, Tiago Miguel Dourado; Silva, Alexandra Maia eFor a diverse range of diseases there are no effective treatments. A refined understanding of the underlying molecular mechanisms of disease and effective targeted therapies are still required for several life-threatening disorders. In the past decade, microRNAs have been discovered as master regulators of thousands of genes at the post-transcriptional level in both normal physiological conditions and in disease. These small non-coding RNAs are highly conserved among animals and their inappropriate expression has been linked to a variety of diseases, such as, cancer, neurodegenerative, autoimmune and cardiovascular diseases. Based on these remarks, miRNA-based therapies are being developed by several pharmaceutical companies with the principle to enhance disease response and elevate cure rates. Therapeutic strategies based on modulation of miRNA expression and function, namely, miRNA blocking and miRNA replacement therapies have been studied in recent years. Once, miRNAs act as key molecules affecting many cellular processes through the regulation of different genes, therapies based on miRNAs are expectable to be particularly effective in heterogeneous diseases that cannot be treated by a single therapeutic agent. However, off-target effects are expected as a result of pleiotropic nature of microRNAs. Despite the fact of many drug discovery programs are ongoing, the most advanced of these programs are yet in phase 2 clinical trials for the treatment of hepatitis C virus infection. An additional effort need to be made to bring these microRNA-based approaches to the clinic.